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Internal Medicine

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Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks


Population:

  • Patients with type 1 or type 2 hereditary angioedema (HAE) aged 12 years or older.

  • Total of 136 participants were enrolled, with 110 participants included in the full analysis population.

Intervention:

  • Oral administration of sebetralstat at doses of 300 mg or 600 mg.

Comparison:

  • Placebo.

Outcome:

  • Primary Outcome:

  • Time to the beginning of symptom relief, defined as a rating of “a little better” on the Patient Global Impression of Change (PGI-C) scale at two or more consecutive time points within 12 hours after the first administration.

  • Results: The median time to the beginning of symptom relief was significantly faster with sebetralstat 300 mg (1.61 hours) and 600 mg (1.79 hours) compared to placebo (6.72 hours) (P < 0.001 and P = 0.001, respectively).

  • Secondary Outcomes:

  • Reduction in Attack Severity:

  • Defined as an improved rating on the Patient Global Impression of Severity (PGI-S) scale at two or more consecutive time points within 12 hours after the first administration.

  • Results: The median time to reduction in attack severity was faster with sebetralstat 300 mg (9.27 hours) and 600 mg (7.75 hours) compared to placebo (more than 12 hours) (P = 0.004 and P = 0.003, respectively).

  • Complete Resolution of the Attack:

  • Defined as a rating of “none” on the PGI-S scale within 24 hours after the first administration.

  • Results: The percentage of attacks with complete resolution within 24 hours was higher with sebetralstat 300 mg (42.5%) and 600 mg (49.5%) compared to placebo (27.4%) (P = 0.002 and P < 0.001, respectively).

  • Safety:

  • The safety profiles of sebetralstat and placebo were similar, with no serious adverse events related to the trial agents reported.


Conclusion: Oral sebetralstat provided faster times to the beginning of symptom relief, reduction in attack severity, and complete attack resolution compared to placebo, with a similar safety profile.


Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks | New England Journal of Medicine (nejm.org)

Harsh Sura
Noor Shah
Manar  Ismail

This would be so beneficial for all the HAE patients, one of the few instances where you fall in love with how medicine is progressing. Hopefully it will be covered by insurance.

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